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EP 3 209 783 A1

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(43) Date of publication: 30.08.2017 Bulletin 2017/35 (21) Application number: 15794644.3 (22) Date of filing: 23.10.2015

(51) International Patent Classification (IPC):  C12N 15/85(2006.01) A61K 38/45(2006.01) A61K 48/00(2006.01) C12N 9/10(2006.01) (86) International application number: PCT/IB2015/058202 (87) International publication number: WO 2016/063264 (28.04.2016 Gazette 2016/17)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA ME Designated Validation States: MA (30) Priority: 24.10.2014 GB 201418965 (71) Applicants: Ospedale San Raffaele S.r.l. 20132 Milano (IT) Fondazione Telethon 00185 Roma (IT)	(72) Inventors: NALDINI, Luigi 20132 Milano (IT) LOMBARDO, Angelo Leone 20132 Milano (IT) AMABILE, Angelo 20132 Milano (IT) MIGLIARA, Alessandro 20132 Milano (IT) (74) Representative: D Young & Co LLP  120 Holborn London EC1N 2DY London EC1N 2DY (GB)